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CureDuchenne, a California-based non-profit organization dedicated to finding a cure for Duchenne Muscular Dystrophy (DMD), announced on Tuesday their $7 million (€5 million) collaboration with the biotechnology company, Prosensa Holding N.V.

The Prosensa-CureDuchenne relationship began in 2004 with the non-profit's $1.3 million investment, a then-unusual case where the venture philanthropy group secured an equity interest in commercial drug development.

The new support, in the form of milestone-linked convertible promissory notes, is intended to accelerate the company's clinical development of RNA-based drugs against the muscle-wasting, childhood disease.

Four Prosensa drugs are currently in various phases of clinical trials, with drisapersen (PRO051) their most advanced.

Nearly 20,000 boys in the U.S., and 300,000 worldwide, suffer from Duchenne muscular dystrophy, the most severe form of a single-gene disorder causing progressive loss of functioning muscle tissue.

As the muscle fiber cells die, they are replaced by fat and less pliable, inactive fibrous tissue. Boys usually become wheelchair-bound by age 12, with a life expectancy rarely outside of the mid-20s as the heart and respiratory muscles lose their capacity.

DMD is often first diagnosed when infants display muscle weakness and young boys first seem to be unable to keep up with their peers. But it can be diagnosed by genetic testing during pregnancy.

First described in the 1830s, Duchenne is named for the French physician who published extensively on the disorder in the 1860s.

Active support of drug development

CureDuchenne was founded in 2003 by business professionals Debra and Paul Miller, parents of a now-17-year-old boy with DMD. CureDuchenne has made strategic investments in several companies developing gene-based treatments to slow the progression of Duchenne.

"Unfortunately, the Duchenne community really isn't, as a group, big enough to cure itself. We can't cure the disease by just staying within the Duchenne families." said Debra Miller, CureDuchenne president. "So, we've had to find ways to be innovative and reach out beyond the Duchenne community to get significant funds."

"We've cured some mice early on and, as we've moved out of the mouse models and into human clinical trials, it's exponentially more expensive. And so, therefore, we have to be more creative and take a different business model that we can get significant funding now," said Miller.

CureDuchenne has also provided support to other companies with related approaches to DMD, such as Sarepta Therapeutics and PTC Therapeutics. Sarepta's eteplirsen targets the same molecular defect as Prosensa's drisapersen. PTC's ataluren (Translarna; PTC124) is a more broadly-acting drug that targets several mutations in DMD and cystic fibrosis using a different molecular mechanism, called translational readthrough, that differs from that of Prosensa and Sarepta.

None of these drugs have been approved in the United States. However, South Plainfield, New Jersey-based PTC Therapeutics received conditional approval from the European Commission on August 4 for ataluren while a confirmatory phase III trial continues. The drug can be used for boys aged five years and up with nonsense mutations in the dystrophin gene in the 28 member states of the EU as well as Iceland, Liechtenstein, and Norway.